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Trial record 3 of 3 for:    Recruiting Studies | Familial Chylomicronemia Syndrome
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Study of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome (FCS) (PALISADE)

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ClinicalTrials.gov Identifier: NCT05089084
Recruitment Status : Recruiting
First Posted : October 22, 2021
Last Update Posted : June 1, 2023
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

Study Description
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Brief Summary:
The purpose or AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of ARO-APOC3 or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive ARO-APOC3.

Condition or disease Intervention/treatment Phase
Familial Chylomicronemia Drug: ARO-APOC3 Drug: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 72 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome
Actual Study Start Date : January 11, 2022
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : April 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Family Issues

Arms and Interventions
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Arm Intervention/treatment
Experimental: ARO-APOC3

4 doses of ARO-APOC3 by subcutaneous (sc) injection (randomized period)

8 doses of ARO-APOC3 by sc injection (open-label period)

 Drug: ARO-APOC3
ARO-APOC3 injection
Placebo Comparator: Placebo
calculated volume to match active treatment by sc injection (randomized period)
 Drug: Placebo
sterile normal saline (0.9% NaCl)


Outcome Measures
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Primary Outcome Measures :
  1. Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10 [ Time Frame: Baseline, Month 10 ]

Secondary Outcome Measures :
  1. Percent Change from Baseline in Fasting TG at Month 10 and Month 12 (Averaged) [ Time Frame: Baseline, Month 10, Month 12 ]
  2. Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10 [ Time Frame: Baseline, Month 10 ]
  3. Percent Change from Baseline in Fasting APOC3 at Month 12 [ Time Frame: Baseline, Month 12 ]
  4. Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10 [ Time Frame: Baseline, Month 10 ]
  5. Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10 [ Time Frame: Baseline, Month 10 ]
  6. Percent Change from Baseline in Fasting TG at Month 12 [ Time Frame: Baseline, Month 12 ]
  7. Percent Change from Baseline in Fasting Non-HDL-C at Month 12 [ Time Frame: Baseline, Month 12 ]
  8. Percent Change from Baseline in Fasting HDL-C at Month 12 [ Time Frame: Baseline, Month 12 ]
  9. Proportion of Patients Achieving TG of < 500 mg/dL at Month 10 [ Time Frame: Month 10 ]
  10. Proportion of Patients Achieving TG of < 500 mg/dL at Month 12 [ Time Frame: Month 12 ]
  11. Change from Baseline in Fasting TG Over Time [ Time Frame: Baseline, up through Month 12 ]
  12. Percent Change from Baseline in Fasting TG Over Time [ Time Frame: Baseline, up through Month 12 ]
  13. Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs) [ Time Frame: From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period) ]
  14. Number of Participants with Positively Adjudicated Events of Acute Pancreatitis [ Time Frame: From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period) ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Fasting TG ≥ 10 mmol/L (≥ 880 mg/dL) at screening refractory to standard lipid lowering therapy
  • Diagnosis of FCS
  • Willing to follow dietary counseling as per investigator judgement based on local standard of care
  • Participants of childbearing potential (males & females) must use highly-effective contraception during the study and for at least 24 weeks following the last dose of study medication. Males must not donate sperm during the study and for at least 24 weeks following the last dose of study medication
  • Women of childbearing potential must have a negative pregnancy test at Screening and cannot be breastfeeding
  • Women of childbearing potential on hormonal contraceptives must be stable on the medication for ≥ 2 menstrual cycles prior to Day 1

Exclusion Criteria:

  • Current use or use within the last 365 Days from Day 1 of any hepatocyte-targeted siRNA or antisense oligonucleotide molecule
  • Diabetes mellitus newly diagnosed within 12 weeks of Screening or where HbA1c ≥ 9.0% at Screening
  • Active pancreatitis within 12 weeks before Day 1
  • History of acute coronary syndrome event within 24 weeks of Day 1
  • History of major surgery within 12 weeks of Day 1
  • Uncontrolled hypertension
  • On treatment with human immunodeficiency virus (HIV) antiretroviral therapy
  • Seropositive for hepatitis B virus (HBV) or hepatitis C virus (HCV)
  • New York Heart Association (NYHA) Clas II, III, or IV heart failure

Note: Additional Inclusion/Exclusion criteria may apply per protocol

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05089084


Contacts
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Contact: Clinical Operations Lead 626-304-3400 AROAPOC@arrowheadpharma.com

Locations
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Sponsors and Collaborators
Arrowhead Pharmaceuticals
More Information
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Responsible Party: Arrowhead Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05089084    
Other Study ID Numbers: AROAPOC3-3001
First Posted: October 22, 2021    Key Record Dates
Last Update Posted: June 1, 2023
Last Verified: May 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type I
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
 Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases